See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as tetrahydrobiopterin, is a medication used to reduce the symptoms of phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine [1]. PKU can cause intellectual disability, behavioral problems, and other complications if left untreated.

According to the US National Library of Medicine, sapropterin can help control the levels of phenylalanine in the blood, which in turn can help manage the symptoms of PKU [2]. However, it's important to note that sapropterin is not a cure for PKU, and it's most effective when used in combination with a phenylalanine-restricted diet.

Specific symptoms that sapropterin has been shown to control include:

1. Elevated phenylalanine levels: Sapropterin can help reduce the levels of phenylalanine in the blood, which is a key goal of PKU treatment [2].
2. Neurological symptoms: PKU can cause neurological symptoms such as seizures, tremors, and developmental delays. Sapropterin has been shown to improve these symptoms in some patients [3].
3. Behavioral symptoms: PKU can also cause behavioral problems such as hyperactivity, aggression, and anxiety. Sapropterin has been shown to improve these symptoms in some patients [3].

It's important to note that sapropterin is not effective for all patients with PKU, and its effectiveness can vary depending on the individual's genetic profile and other factors [2].

Sources:

1. DrugPatentWatch.com. (n.d.). Sapropterin Dihydrochloride. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride>.
2. US National Library of Medicine. (2019). Sapropterin. Retrieved from <https://medlineplus.gov/druginfo/meds/a613042.html>.
3. National PKU Alliance. (2021). Sapropterin (Kuvan). Retrieved from <https://www.npkua.org/living-with-pku/treatment/sapropterin-kuvan/>.